OrphanDC
Fostering Innovation and Clinical Development in Latin America

In the face of growing global competitiveness in clinical trial patient enrollment, combined with the political pressures of Eastern Europe, Latin America has been making significant strides in becoming a key contributor to the global biotechnology industry. With a heterogeneous population, the region is poised to ascend to the upper echelon of the global clinical trial market. A pivotal contributor to this impressive development is OrphanDC, a biotech firm that has emerged as a trusted partner for its Latin American counterparts.

“OrphanDC is committed to establishing and creating the necessary infrastructure to help build Latin America’s own homegrown life sciences ecosystem”, says Daniel Zequeto, Founder and Managing Partner at OrphanDC.

OrphanDC’s suite of services encompasses hosting, early access, clinical trials, and distribution services, all designed to cater to the unique needs of biotech firms in the region. The company, with its team of industry veterans, specializes in providing clinical development services for innovative biotechnology companies, aiming to facilitate the drug’s entire life cycle for its global clients.

The firm has been instrumental in supporting and facilitating innovation on a global scale. It has helped generate clinical data recently used in Apellis Pharmaceutical’s Syfovre. Additionally, the company has collaborated with innovative companies like Agenus, Global Blood Therapeutics, and Albireo Pharma. This organization stands apart from its competitors by acknowledging Latin America as a comprehensive destination for conducting clinical trials rather than solely as a region for patient recruitment.

To underscore its core competencies, a remarkable success story can be seen in its partnership with Apellis not only for Syfovre but also in the relationship that began when OrphanDC seamlessly recruited patients with Paroxysmal nocturnal hemoglobinuria (PNH) for both their Phase Two and Phase Three clinical trials.

In the face of stiff competition from the commercialization effort by Alexion’s Soliris, OrphanDC supported Apellis in expeditiously recruiting patients within stringent timelines. It facilitated the mapping of patient flow, timely follow-up with each enrolled patient, and offered unwavering assistance throughout the journey from enrollment to trial completion. Ultimately, Apellis utilized the data generated from the trial to register and obtain FDA approval for its drug.

OrphanDC is a hybrid entity comprising two distinct divisions - OrphanDC CT, which specializes in clinical development and CRO services, and OrphanDC G, which concentrates on business commercialization and facilitating early patient access. OrphanDC CT provides customized clinical development services, starting with a tailor-made feasibility and environmental assessment that streamlines trial processes. It identifies the unique regional characteristics of all stakeholders to maximize the potential of the trials conducted. Moreover, OrphanDC CT also functions as a full-service patient support and travel management program that is a concierge for patient travel arrangements, including passports and visas.

In clinical research monitoring services, OrphanDC CT offers local expertise from the pre-feasibility stage to foster site engagement and qualitative patient enrollment while minimizing screening errors. It utilizes a multilayered site support strategy and actively participates in clinical team training and other crucial research-related tasks. Similarly, OrphanDC CT upholds a rigorous guideline-abiding framework that governs smooth site payments and budgeting. In addition, OrphanDC CT maintains an in-house safety and pharmacovigilance department that oversees ethical submissions and follow-up meetings to ensure the safety of its services.

Shifting focus to OrphanDC G, its mission is to support commercial business operations and market access through a multidisciplinary team comprising back-office and fieldwork support. It seeks to assist stakeholders through all essential steps in the patient’s journey to access treatment. Furthermore, the company imports and manages pharmaceutical products across Latin America, navigating complicated regulatory guidelines with expert knowledge and key industry relationships.

In pursuit of its noble goal to bring innovative medicines to the people of Latin America, OrphanDC G bridges the gap between patients and cutting-edge investigational drugs that fall outside the ambit of clinical trials. The company forges formidable bonds between patients and the medical community by leveraging the Named Patient Process (NPP) that empowers patients and medical professionals to access foreign-approved drugs in their respective nations.

With facilities in Brazil, Argentina, and Colombia, the company endeavors to catalyze Latin America’s healthcare sector, thus propelling it to newfangled heights, especially in light of the inherent weaknesses of more traditional markets.